Key Takeaways RCKT received FDA clearance to start clinical trials for its gene therapy candidate RP-A701. The phase I study will assess RP-A701's safety, biological activity and preliminary efficacy. Participants will receive a single RP-A701 dose, with multiple cardiac health markers being monitored.Rocket Pharmaceuticals (RCKT) announced that the FDA has cleared its investigational new drug (IND) application seeking to begin clinical studies on its first-in-class gene therapy candidate, RP-A701.The com ...

Shares of Rocket Pharmaceuticals (RCKT) plunged 62.8% on Tuesday after the company announced a patient death in a pivotal phase II study evaluating its investigational gene therapy candidate, RP-A501, for treating patients with Danon disease.The company confirmed that one of the patients in the study experienced an acute systemic infection, an unexpected serious adverse event, which resulted in his untimely death.Following the turn of events, RCKT has voluntarily paused further dosing in the study evaluatin ...

Financial Performance - Rocket Pharmaceuticals incurred a loss of 56 cents per share in Q1 2025, which is narrower than the Zacks Consensus Estimate of a loss of 59 cents and an improvement from a loss of 66 cents per share in the same quarter last year [1] - The company did not record any revenues in the reported quarter, missing the Zacks Consensus Estimate for total revenues of $8 million [2] - General and administrative expenses rose 28% year over year to $28.4 million, attributed to increased commercial preparation and legal expenses [2] - Research and development expenses were $35.9 million, down 21% from the previous year due to reduced manufacturing and development costs [3] - As of March 31, 2025, the company had cash, cash equivalents, and investments totaling $318.2 million, down from $372.3 million as of December 31, 2024, with expectations to fund operations into Q4 2026 [3] - Year to date, RCKT shares have declined 44%, compared to an 8% decline in the industry [4] Pipeline Developments - Kresladi, developed for treating severe leukocyte adhesion deficiency-I (LAD-I), received a complete response letter (CRL) from the FDA in June 2024, requesting limited additional information on the Chemistry Manufacturing and Controls (CMC) [7][8] - The company plans to file a complete BLA to resolve the CRL later in 2025 [8] - Rocket Pharmaceuticals is developing RP-L102 for treating Fanconi anemia (FA) and has initiated a rolling BLA, expecting to complete the submission in late 2025 or early 2026 [9] - Dosing is currently underway in a phase II pivotal study for RP-A501, targeting male patients with Danon disease, with clinical data readout expected in mid-2026 [10]